Continuation of reforms to TGA and PBS to improve access to new medicines for Australians with blood cancers.

The Issue

The Leukaemia Foundation is concerned that our regulator of new medicines (TGA) and medicines access scheme (PBS) continue to evolve
to remain fit for purpose in the age of new genetics-based, targeted medicines
for specific disease subtypes.

What we know

Our understanding of cancer is changing rapidly and improving
significantly our ability to identify, name and treat cancer by its genetic
characteristics. Increasingly cancers are being recognised as subtypes of
disease rather than simply the organ in which they occur.

The genetic features of subtypes are being used as targets for
the development of new and specific medicines – called personalised medicine or
precision medicine.

Patients welcome precision medicines as it means that they are
able to receive a specific treatment developed for their particular blood
cancer subtype and avoid heavy duty chemo with its immediate and ongoing
detrimental side effects.

However, subtype therapies can magnify the challenges of
obtaining the quality clinical data required to demonstrate cost effectiveness
to the PBAC for rare subtypes of already rare blood cancers.

In the last 10 years, there have been around 20 new blood
cancer therapies added to the PBS (Medicines
Australia Facts Book 2) and about 300 therapies
are under development (PhRMA, 2015).

The Leukaemia Foundation is concerned that the current PBS
system in Australia will not effectively cope with the number of new generation
treatments.

Blood cancer patients often express concern that drugs made
available on the PBS for the common cancers, exclude people with rare forms of
cancer, even if their cancer has the same genetic target.

The Leukaemia Foundation believes that there should be a score
allocated for patient-reported quality of life as part of PBAC assessment.

The PBAC’s ability to assess national treatment outcome data
would be significantly enhanced with a national clinical quality blood cancer
registry. Such registries enable the evaluation of the appropriateness and
effectiveness of treatments and other interventions in the healthcare (ACSQHC,
2014).

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